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1 products found

pp-149

Solution for injection 80 mg/1.5 ml

Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Severe hypertriglyceridaemia (sHTG; TG ≥500 mg/dL); familial chylomicronaemia syndrome (FCS, approved Tryngolza) Clinical stage: Phase 3 Modality: Biologic/oligonucleotide (GalNAc-conjugated antisense oligonucleotide, ASO) Mechanism / Target: Targets hepatic APOC3 mRNA — reduces apolipoprotein C-III production, increasing LPL-dependent and LPL-independent clearance of TG-rich lipoproteins Route / form: Subcutaneous injection (monthly) Differentiation: Monthly SC dosing; liver-selective GalNAc targeting; superior TG reduction vs fibrates/omega-3; first APOC3 ASO approved for FCS; expanding to broader sHTG population
Manufacturer #39180

Leader in RNA-targeting therapeutics with multiple approved antisense drugs. Develops GalNAc-conjugated ASOs for precise liver-targeted gene silencing in cardiometabolic and rare diseases. Wholly owned commercial and late-stage pipeline with independent global development.
 

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